scholarly journals Current Trends in the Gene Therapy of Hematologic Disorders

2021 ◽  
Vol 48 (4) ◽  
pp. 50-56
Author(s):  
D. Nikolova

Abstract Recent advances in molecular genetics and the invention of new technologies led to an advance in the development of gene therapy. Gene therapy is used to correct defective genes in order to cure a disease or help the body better fight a disease. It works by restoring or modifying cellular functions through the introduction of a functional gene into the target cell. The concept of gene therapy is simple, but introducing it to routine clinical practice is not. The main concerns are related to some safety issues as well as to the problem that maintaining a stable and prolonged expression in target cells may not be easily achieved. In spite of the difficulties, gene therapy remains a hope for many hematological disorders that cannot be effectively treated so far. This article reviews the current status of gene therapy with a focus on hematological disorders. In addition, clinically applied approaches are presented through particular examples of approved gene therapy drugs.

Author(s):  
Reetu Malik ◽  
Jyoti Rathi ◽  
Deeksha Manchanda ◽  
Manish Makhija ◽  
Deepshikha Kushwaha ◽  
...  

Background: The safety and quality of food has been a matter of great discussion throughout the centuries and the application of nanotechnology in the field of nutraceuticals i.e. nanoceuticals has improved the variety and protection of food products in many ways. Objective: Improving the quality and safety of the food products with a view to improve public health and the invasion of nanotechnological advancements in the area of nutrition has resulted in the expansion of novel foods with improved oral bioavailability as well as thermal stability. The main objective of this review is to summarize available literature on nanoceuticals including patents and clinical trials. Method: The review was extracted from the searches performed at PubMed, Google Patents and Google Scholars, etc. Data from these searches was collected and evaluated for getting the information about the available literature on the nanoceuticals. Along with this, some reported patents have also been included in this review in order to conclude the future of nanoceuticals. Result: The literature so obtained was studied thoroughly as per the requirement of the objective of this review. The details of nanoceuticals including major applications, regulatory aspects, some reported patents and clinical trials are compiled here in this review. Nanoceuticals like vitamins, antibiotics, bioactive peptides, probiotics etc., which are dispersed, absorbed or incorporated in nano diameter range sacs, having improved solubility, delivery properties, biological activities, protection against degradation and therefore having improved biological activities and delivery to the target cells and tissues in the body. Different regulations from various countries recommended that any food ingredients which results from the use of nanotechnologies must undergo safety risk assessment standards before entering into the market as nano-food. Conclusion: The idea of “nanoceuticals” is increasing enthusiasm and marketable dairy/food and food supplements. This article focuses on the history, applications, regulation aspects, patents, clinical trials and future prospects of nanoceuticals.


2019 ◽  
Vol 7 (3) ◽  
pp. 220-233 ◽  
Author(s):  
Shrestha Sharma ◽  
Md. Noushad Javed ◽  
Faheem Hyder Pottoo ◽  
Syed Arman Rabbani ◽  
Md. Abul Barkat ◽  
...  

The traditional drug delivery techniques are unresponsive to the altering metabolic states of the body and fail to achieve target specific drug delivery, which results in toxic plasma concentrations. In order to harmonize the drug release profiles, diverse biological and pathological pathways and factors involved have been studied and consequently, nanomaterials and nanostructures are engineered in a manner so that they respond and interact with the target cells and tissues in a controlled manner to induce promising pharmacological responses with least undesirable effects. The bioinspired nanoparticles such as carbon nanotubes, metallic nanoparticles, and quantum dots sense the localized host environment for diagnosis and treatment of pathological states. These biocompatible polymeric- based nanostructures bind drugs to the specific receptors, which renders them as ideal vehicles for the delivery of drugs and gene. The ultimate goal of bioinspired nanocomposites is to achieve personalized diagnostic and therapeutic outcomes. This review briefly discussed current trends; role, recent advancements as well as different approaches, which are being used for designing and fabrication of some bioinspired nanocarriers.


Author(s):  
Wesley Wierson ◽  
Alex Abel ◽  
Elizabeth Siegler ◽  
Stephen Ekker ◽  
Chad Johannes ◽  
...  

With rapid advances in gene editing and gene therapy technologies, the development of genetic, cell, or protein-based cures to disease are no longer the realm of science fiction but that of today’s practice. The impact of these technologies are rapidly bringing them to the veterinary market as both enhanced therapeutics and towards modeling their outcomes for translational application. Simply put, gene editing enables scientists to modify an organism’s DNA a priori through the use of site-specific DNA targeting tools like clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9). Gene therapy is a broader definition that encompasses the addition of exogenous genetic materials into specific cells to correct a genetic defect. More precisely, the U.S Food and Drug Administration (FDA) defines gene therapy as “a technique that modifies a person’s genes to treat or cure disease” by either (i) replacing a disease-causing gene with a healthy copy of the gene; (ii) inactivating a disease-causing gene that was not functioning properly; or (iii) introducing a new or modified gene into the body to help treat a disease. In some instances, this can be accomplished through direct transfer of DNA or RNA into target cells of interest or more broadly through gene editing. While gene therapy is possible through the simple addition of genetic information into cells of interest, gene editing allows the genome to be reprogrammed intentionally through the deletion of diseased alleles, reconstitution of wild type sequence, or targeted integration of exogenous DNA to impart new function. Cells can be removed from the body, altered, and reinfused, or edited in vivo. Indeed, manufacturing and production efficiencies in gene editing and gene therapy in the 21st century has brought the therapeutic potential of in vitro and in vivo reprogrammed cells, to the front lines of therapeutic intervention (Brooks et al., 2016). For example, CAR-T cell therapy is revolutionizing hematologic cancer care in humans and is being translated to canines by us and others, and gene therapy trials are ongoing for mitral valve disease in dogs.


Author(s):  
Anastasiya G. Khotuleva ◽  
Mariya S. Kozyreva

Introduction. The most susceptible to lead is the hematopoietic system of hematopoietic organs due to lead inhibition of heme and globin synthesis and cytotoxic effect on the membrane of Mature red blood cells. The aim of study was to evaluate the informative value of the study of erythrocyte and reticulocyte parameters determined on modern hematological analyzers in patients working in contact with lead during medical and biological monitoring. Materials and methods. 45 employees of the lead battery processing plant and 30 persons of control group were examined. The level of lead in the blood was determined by atomic absorption spectrometry, δ-ALA in the urine-by the reaction of pyrol formation with acetylacetone in terms of gram of creatinine, the study of hematological parameters was performed on a Sysmex HT-2000i analyzer. Statistical processing of the results was performed using the program STATISTICA 10.0. Results. Significant changes in erythrocytic (RDW) and reticulocytic (RET, IRF, LFR, MFR, HFR, RET-He) parameters, erythropoietin in workers in contact with lead compared to the control group, changes in MCV, MCH, RDW, RET indicators in the group working in dynamics after 2 years were revealed. Associations of hematological parameters with biomarkers of exposure and effect (lead level in blood and ALA in urine) were revealed. Conclusions. Assessment of erythrocyte (MCV, MCH, RDW) and reticulocyte parameters (RET% and their distribution by maturity) in dynamics during periodic medical examinations of workers in contact with lead allows us to detect the development of hematological disorders at early stages.


Author(s):  
Kinesh V P ◽  
Neelam D P ◽  
Punit B ◽  
Bhavesh S.B ◽  
Pragna K. S

Diabetes mellitus is a serious pathologic condition that is responsible for major healthcare problems worldwide and costing billions of dollars annually. Insulin replacement therapy has been used in the clinical management of diabetes mellitus for more than 84 years. The present mode of insulin administration is by the subcutaneous route through which insulin is presented to the body in a non-physiological manner having many challenges. Hence novel approaches for insulin delivery are being explored. Challenges to oral route of insulin administration are: rapid enzymatic degradation in the stomach, inactivation and digestion by proteolytic enzymes in the intestinal lumen and poor permeability across intestinal epithelium because of its high molecular weight and lack of lipophilicity. Liposomes, microemulsions, nanocubicles, and so forth have been prepared for the oral delivery of insulin. Chitosan-coated microparticles protected insulin from the gastric environment of the body and released intestinal pH. Limitations to the delivery of insulin have not resulted in fruitful results to date and there is still a need to prepare newer delivery systems, which can produce dose-dependent and reproducible effects, in addition to increased bioavailability.


2018 ◽  
Vol 9 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Aparna Bansal ◽  
Himanshu

Introduction: Gene therapy has emerged out as a promising therapeutic pave for the treatment of genetic and acquired diseases. Gene transfection into target cells using naked DNA is a simple and safe approach which has been further improved by combining vectors or gene carriers. Both viral and non-viral approaches have achieved a milestone to establish this technique, but non-viral approaches have attained a significant attention because of their favourable properties like less immunotoxicity and biosafety, easy to produce with versatile surface modifications, etc. Literature is rich in evidences which revealed that undoubtedly, non–viral vectors have acquired a unique place in gene therapy but still there are number of challenges which are to be overcome to increase their effectiveness and prove them ideal gene vectors. Conclusion: To date, tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors. This review mainly summarizes the various physical and chemical methods for gene transfer in vitro and in vivo.


2021 ◽  
pp. 097168582110159
Author(s):  
Sital Mohanty ◽  
Subhasis Sahoo ◽  
Pranay Kumar Swain

Science, technology and human values have been the subject of enquiry in the last few years for social scientists and eventually the relationship between science and gender is the subject of an ongoing debate. This is due to the event of globalization which led to the exponential growth of new technologies like assisted reproductive technology (ART). ART, one of the most iconic technological innovations of the twentieth century, has become increasingly a normal social fact of life. Since ART invades multiple human discourses—thereby transforming culture, society and politics—it is important what is sociological about ART as well as what is biological. This article argues in commendation of sociology of technology, which is alert to its democratic potential but does not concurrently conceal the historical and continuing role of technology in legitimizing gender discrimination. The article draws the empirical insights from local articulations (i.e., Odisha state in eastern India) for the understandings of motherhood, freedom and choice, reproductive right and rights over the body to which ART has contributed. Sociologically, the article has been supplemented within the broader perspectives of determinism, compatibilism alongside feminism.


2021 ◽  
Vol 9 (4) ◽  
pp. 868
Author(s):  
Max Maurin ◽  
Florence Fenollar ◽  
Oleg Mediannikov ◽  
Bernard Davoust ◽  
Christian Devaux ◽  
...  

SARS-CoV-2 is currently considered to have emerged from a bat coronavirus reservoir. However, the real natural cycle of this virus remains to be elucidated. Moreover, the COVID-19 pandemic has led to novel opportunities for SARS-CoV-2 transmission between humans and susceptible animal species. In silico and in vitro evaluation of the interactions between the SARS-CoV-2 spike protein and eucaryotic angiotensin-converting enzyme 2 (ACE2) receptor have tentatively predicted susceptibility to SARS-CoV-2 infection of several animal species. Although useful, these data do not always correlate with in vivo data obtained in experimental models or during natural infections. Other host biological properties may intervene such as the body temperature, level of receptor expression, co-receptor, restriction factors, and genetic background. The spread of SARS-CoV-2 also depends on the extent and duration of viral shedding in the infected host as well as population density and behaviour (group living and grooming). Overall, current data indicate that the most at-risk interactions between humans and animals for COVID-19 infection are those involving certain mustelids (such as minks and ferrets), rodents (such as hamsters), lagomorphs (especially rabbits), and felines (including cats). Therefore, special attention should be paid to the risk of SARS-CoV-2 infection associated with pets.


2018 ◽  
Vol 08 (04) ◽  
pp. 024-027
Author(s):  
Shiny Inasu ◽  
Biju Thomas

AbstractA novel noninvasive photochemical approach for infection control, namely photodynamic therapy, has received much attention in the treatment of oral diseases which requires three nontoxic ingredients namely visible harmless light, a photosensitizer and oxygen are involved in this therapy. It is based on the principle that a photosensitizer binds to the target cells which when activated by light of a suitable wavelength results in the production of singlet oxygen and other very reactive agents that are extremely toxic to certain cells and bacteria. This article highlights the application of photo-dynamic therapy in management of periodontal disease and its current status.


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